By adjusting the levels of mTORC1, a key signaling protein in the brain, researchers improved motor function and brain abnormalities in experimental animals with a form of Huntington's disease.The study leader Dr. Beverly L. Davidson, director of The Center for Cellular and Molecular Therapeutics at The Children's Hospital of Philadelphia (CHOP) is hopeful that results in animals can open the door to a potential therapy, based on carefully manipulating the dysregulated pathway to treat this disease in humans. She added that restoring proper balance to these delicate biological processes may offer even broader benefits in treating other neurological diseases, such as amyotrophic lateral sclerosis (ALS), fragile X mental retardation and autism.Neuroscientists already knew that a signaling protein called mTORC1 that regulates cell growth and metabolism plays a major role in HD. Many researchers have proposed that inhibiting or shutting off the mTORC1 pathway, which interacts with the deleterious mHTT proteins, could help treat HD.The current study contradicts those assumptions. "We show that the mTORC1 pathway is already impaired in Huntington's disease, and that improving how the pathway functions actually has a protective effect," said Davidson. "However, restoring that pathway must be done very carefully to avoid further harm. It's a 'Goldilocks effect.' You need to restore the mTORC1 level; either too much or too little is detrimental."